A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Open to males ages 10 years and up

1. Male subjects at least 10 years of age at time of consent who are willing and able to provide informed consent to participate in the trial if ≥ 18 years of age or assent with parental or guardian informed consent if < 18 years of age. If a third-party caregiver is involved, they must provide informed consent.
2. Diagnosis of DMD based on clinical and phenotypic manifestations consistent with DMD (e.g., family history of DMD, elevated creatine kinase, dystrophin muscle biopsy, calf pseudohypertrophy, history of Gowers' sign, and gait impairment before 7 years of age) as confirmed by the Investigator.
3. Confirmatory genetic testing performed to have reached a diagnosis of DMD at any time in the past or currently performed at a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory or equivalent.
4. Performance of the Upper Limb test (PUL) entry item scores 2-6 and total PUL score less than or equal to 40. For Cohort A only: enrollment of patients with PUL entry score 6, Exon 44 skipping amenable, and/or Exon 3 through 7 deletions will be capped at no more than 10% of the total study population (approximately 6 patients with these characteristics).
5. Reduced ability to walk/run (if ambulatory): subjects must take more than 10 seconds for the 10-meter walk/run (i.e., velocity < 1 meter/second).
6. If non-ambulatory, loss of independent ambulation between 10th and 18th year birthday (standing unassisted or ability to take, at most, several steps independently is not considered ambulation). Subjects who are considered non-ambulatory between the ages of 9 and10 may be enrolled with prior approval from the sponsor.
7. Receiving standard of care therapy at an experienced, multidisciplinary DMD center as evidenced by regular cardiac and pulmonary monitoring, systemic glucocorticoid treatment, and at-home range of motion exercises.
8. Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least 6 months prior to study participation, except for either weight-based dose adjustment or a decrease in steroid dose of ≤ 10% for toxicity. For patients on chronic deflazacort, treatment with an equivalent dose of prednisone or prednisolone for a period of ≤ 30 days to bridge lack of availability of deflazacort during the 6 months prior to randomization is acceptable.

9. Current and up-to-date immunizations according to children and adolescent Centers for Disease Control and Prevention immunization schedule at the discretion of the Investigator.

   10. Adequate venous access for parenteral IP infusions and routine blood collection. 11. Assessed by the Investigator as willing and able to comply with the requirements of

   the trial.

   12. Sexually active subjects and their partners who are fertile must agree to use

   effective method(s) of contraception.

1. Left ventricular ejection fraction (LVEF) less than or equal to 35% prior to randomization.
2. Elbow-flexion contractures > 30° in both extremities.
3. Body mass index (BMI) > 45.
4. Percent predicted forced vital capacity (FVC%) < 35% within 6 months prior to randomization.
5. Inability to perform consistent PUL 2.0 measurement within ± 2 points without shoulder domain or within ± 3 points with shoulder domain during paired testing at screening.
6. Risk of near-term respiratory decompensation in the judgment of the Investigator, or the need for initiation of day and night non-invasive ventilator support as defined by serum bicarbonate ≥ 29 mmol/L at screening.
7. History of non DMD-related chronic respiratory disease requiring ongoing or intermittent treatment, including, but not limited to, asthma, bronchitis, and tuberculosis.
8. Acute respiratory illness within 30 days prior to screening and during screening.

9. Initiation of nocturnal non-invasive ventilation within 30 days prior to screening.

   10. Planned or anticipated thoracic or spinal surgery within the 6 months following

   randomization.

   11. Planned or anticipated lower extremity surgery within the 6 months following

   randomization, if ambulatory.

   12. Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products. 13. Initiation of treatment with metformin or insulin within 3 months prior to

   randomization.

   14. Initiation of treatment with an FDA-approved exon skipping therapy for the treatment

   of DMD and/or non-weight based adjustments within 12 months prior to randomization.

   15. Treatment with human growth hormone within 3 months prior to randomization, unless on

   a stable dose allowing for weight-based dose adjustments (as determined by the site Investigator) for at least 24 months prior to randomization.

   16. Treatment with a cell therapy product within 12 months prior to randomization; any

   prior exposure to CAP-1002 will be excluded.

   17. Treatment with an investigational product within 6 months prior to randomization. 18. History, or current use, of drugs or alcohol that could impair the ability to comply

   with participation in the trial.

   19. Inability to comply with the investigational plan and follow-up visit schedule for any

   reason, in the judgment of the investigator.

   20. Inability to undergo a cardiac MRI. For Cohort B Only - Subjects with a known

   hypersensitivity to gadolinium may forgo the LGE assessment but must complete a cardiac MRI without contrast. For Cohort B Only - Subjects who are unable to tolerate gadolinium due to renal insufficiency as measured by an estimated Glomerular Filtration Rate (eGFR) less than 60 mL/min/1.73 m2 may forgo the LGE assessment but must complete a cardiac MRI without contrast.

   21. For Cohort B: Subjects with PUL entry score 6, Exon 44 skipping amenable, or Exon 3

   through 7 deletions are excluded from participation.